Translate Bio Announces Oral Presentation of mRNA Therapeutics in Models of Metabolic Disorders at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting
“We have engineered our mRNA therapeutic (MRT) platform to deliver mRNA that codes for the missing enzymes in several metabolic disorders,” said Dr. DeRosa. “These data demonstrate the potential broad applicability of the MRT platform to treat various genetic disorders using our proprietary liver delivery technology.”
The presentation summarizes findings from experiments in three mouse models of metabolic disorders including ornithine transcarbamylase (OTC) deficiency, argininosuccinate synthetase (ASS1) deficiency (citrullinemia) and methylmalonic acidemia (MMA), which are each caused by a deficiency of a single enzyme specific for each disease. Messenger RNA encoding human OTC, human ASS1 or human methylmalonyl-CoA mutase enzymes were synthesized, incorporated in lipid nanoparticles (LNPs) and formulated for intravenous (IV) administration. Data from these studies demonstrated that the mRNA encapsulated in an LNP can be successfully delivered to the liver by IV administration, resulting in the production of the desired enzyme. In each disease model, the enzyme was functional as seen by the reduction, prevention or normalization of the metabolic abnormalities characteristic of these three disorders.
Inherited metabolic disorders are a class of rare diseases with high unmet medical need. Three such diseases were addressed in the ASGCT presentation. OTC deficiency and citrullinemia are inherited UCDs that cause toxic levels of ammonia to accumulate in the blood. MMA is an inherited OA disorder that disrupts normal amino acid metabolism and causes buildup of toxic levels of methylmalonic acid in the blood. High blood levels of these substances can lead to neurological conditions.
The full abstract #26 can be found on the ASGCT conference website at https://www.asgct.org/global/documents/asgct19_abstracts_-final.
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include, but are not limited to, those regarding: the potential for mRNA therapeutics to treat metabolic disorders; the potential benefits of mRNA therapy; Translate Bio’s beliefs regarding the broad applicability of its MRT platform; and Translate Bio’s plans, strategies and prospects for its business, including its lead development programs. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from current expectations and beliefs, including but not limited to: Translate Bio’s ability to advance the development of its platform and programs under the timelines it projects, demonstrate the requisite safety and efficacy of its product candidates and replicate in clinical trials any positive findings from preclinical studies; the content and timing of decisions made by the
Source: Translate Bio, Inc.